FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease

State-of-the-art gene therapy provides new treatment option for children 2 years and older with life-threatening blood disorder Silver Spring, MD, July 01, 2026 (GLOBE NEWSWIRE) -- The U.S. Food and Drug Administration today issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β thalassemia (TDT). This is the first gene therapy approved f